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OwnerCatherine Pachuk, C. Satishchandran +1inventors
InventorsCatherine Pachuk, C. Satishchandran, Maninder Chopra
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The present invention relates to novel double stranded RNA (dsRNA) structures and dsRNA expression constructs, methods for generating them, and methods of utilizing them for silencing genes. Desirably, these methods specifically inhibit the expression of one or more target genes in a cell or animal (e.g., a mammal such as a human) without inducing toxicity. These methods can be used to prevent or treat a disease or infection by silencing a gene associated with the disease or infection. The invention also provides methods for identifying nucleic acid sequences that modulate a detectable phenotype, such as the function of a cell, the expression of a gene, or the biological activity of a target polypeptide.
A nucleic acid molecule that comprises or that encodes, in 5′ to 3′ order, a first region of interest, a first base-paired region, a loop region, and a second base-paired region, wherein said first and second base-paired regions are capable of base-pairing to each other or are base-paired to each other.
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A pharmaceutical composition comprising a vector construct comprising, at the 5′ end, a promoter that is operably linked to a nucleic acid molecule and which enables transcription of said nucleic acid molecule, wherein said nucleic acid molecule encodes, in 5′ to 3′ order, a first region of interest, a first base-paired region, a loop region, and a second base-paired region, wherein said first and second base-paired regions are capable of base-pairing to each other, and wherein transcription of said nucleic acid molecule produces an RNA hairpin.
A method for generating an RNA hairpin comprising transcribing a nucleic acid molecule in a host cell that has been transformed with said nucleic acid molecule, wherein said nucleic acid molecule comprises, at the 5′ end, a promoter that is operably linked to said nucleic acid molecule and which enables transcription of said nucleic acid molecule, and wherein said nucleic acid molecule encodes, in 5′ to 3′ order, a first region of interest, a first base-paired region, a loop region, and a second base-paired region, wherein said first and second base-paired regions are capable of base-pairing to each other, wherein transcription of said nucleic acid molecule produces an RNA hairpin.
A method for inhibiting the expression of a target gene in a cell, said method comprising administering to a subject in need thereof, a nucleic acid molecule that comprises or that encodes an RNA hairpin, wherein said RNA hairpin comprises, in 5′ to 3′ order, a first region of interest, a first base-paired region, a loop region, a second base-paired region, and a second region of interest, wherein said first and second base-paired regions are capable of base-pairing to each other or are base-paired to each other, and wherein said administering inhibits or reduces expression of a target gene, relative to expression of said target gene in a subject not administered said nucleic acid molecule.
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Dependencies are inferred from the claim text.